The Child-Centered Outcomes in Practice and Research (COPR) Center of Excellence: Strengthening the Clinical Validity Evidence Base for PROMIS Measures in Chronically Ill Children

以儿童为中心的实践与研究成果 (COPR) 卓越中心：加强慢性病儿童 PROMIS 措施的临床有效性证据基础

• 批准号: 9077845
• 负责人: CHRISTOPHER B FORREST
• 金额: $ 417万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-09-30 至 2021-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9077845
• 关键词: Address; Adoption; Affect; Centers of Research Excellence; Child; Child health care; Childhood; Chronic; Chronic Disease; Chronic Kidney Failure; Chronically Ill; Clinical; Clinical Data; Clinical Trials; Cohort Studies; Collaborations; Communication; Complement; Copper; Crohn' s disease; Data Coordinating Center; Data Reporting; Device or Instrument Development; Electronic Health Record; Evaluation; Fatigue; Funding; Funding Opportunities; Gastroenterologist; Goals; Happiness; Health; Individual; Informatics; Information Systems; Lead; Life Cycle Stages; Link; Malignant Neoplasms; Measurement; Measures; Methods; Outcome; Outcome Assessment; Outcomes Research; Pain interference; Patient Outcomes Assessments; Patients; Pediatric Hospitals; Pediatric Oncology; Personal Satisfaction; Philadelphia; Psychological Stress; Psychometrics; Qualifying; Reporting; Research; Research Infrastructure; Research Project Grants; Resource Informatics; Resources; Saint Jude Children' s Research Hospital; Science; Standardization; United States National Institutes of Health; Validation; base; clinical practice; cohort; data management; design; evidence base; experience; instrument; pediatric patients; programs; prospective; public health relevance; research in practice; research to practice; response

 DESCRIPTION (provided by applicant): Patient-reported outcome (PROs) instruments complement conventional clinical outcomes by examining from the perspective of patients how they feel, what they perceive they can do, which types of activities they believe they can participate in, and how satisfied they are with their health and lives. To advance the science of PRO assessment, the NIH launched in 2004 the Patient Reported Outcome Measurement Information System (PROMIS(r)). Although a key objective of PROMIS is to provide PROs for research and clinical practice across the life course, there is remarkably little known about the validity of PROMIS for children with chronic diseases or the degree to which PROMIS detects clinically meaningful changes in children's health. To address these scientific needs, we propose to create the Child-Centered Outcomes in Practice and Research (COPR) Center of Excellence, which will be devoted to rigorously examining and documenting PROMIS' clinical validity for children with chronic conditions. COPR comprises three research projects focusing on pediatric Crohn's disease, chronic kidney disease, and cancer. The projects are linked by common measures of children's Experienced Well-Being (a concept that subsumes both suffering and happiness), clinical validation methods, and infrastructure. Each project will assess the content validity, clinical meaningfulness of scores, responsiveness to clinical change, and longitudinal construct validity of the PROMIS Pediatric pain interference, fatigue, psychological stress experiences, and positive affect measures. The COPR approach will increase the measures' psychometric and clinical maturation according to PROMIS standards and provide evidence required by the FDA to qualify PRO instruments for clinical trials. The COPR team integrates: a PROMIS research group that developed 10 of the 20 extant PROMIS pediatric measures and helped lead the PROMIS initiative over the past 6 years; a PCORI-funded clinical data research network (PEDSnet) with deep expertise in electronic health record and PRO data management, quality assessment and reporting; a national network of pediatric gastroenterologists embarking on a PCORI-funded clinical trial for children with Crohn's disease; a national network of pediatric nephrologists who are conducting an NIH-funded cohort study on children with chronic kidney disease; and, one of the nation's largest pediatric oncology clinical centers located at St. Jude Children's Research Hospital. The COPR infrastructure will: facilitate coordination across its three research projects; standardize clinicl validation methods and informatics resources; synthesize results across the three projects; develop standards-based instrument dossiers to enhance clinical adoption of PROMIS and facilitate FDA qualification of PROMIS measures as clinical trial end-points; and, support collaboration with other PEPR research programs. COPR is optimized to successfully accomplish its three research projects, and its infrastructure can be readily scaled to support the informatics, statistical, and administrative needs of multiple other PEPR research initiatives.

 描述（由申请人提供）：患者报告结局（PRO）工具通过从患者的角度检查他们的感受、他们认为他们可以做什么、他们认为他们可以参与哪些类型的活动以及他们对自己的健康和生活的满意度来补充传统的临床结局。为了推进PRO评估的科学，NIH于2004年推出了患者报告结果测量信息系统（PROMIS（r））。虽然PROMIS的一个关键目标是为整个生命过程的研究和临床实践提供PRO，但对于PROMIS对慢性病儿童的有效性或PROMIS检测儿童健康的临床意义变化的程度知之甚少。为了满足这些科学需求，我们建议创建以儿童为中心的实践和研究成果（COPR）卓越中心，该中心将致力于严格检查和记录PROMIS对慢性病儿童的临床有效性。COPR包括三个研究项目，重点是儿科克罗恩病，慢性肾脏疾病和癌症。这些项目通过儿童体验幸福的共同措施（一个包含痛苦和幸福的概念），临床验证方法和基础设施联系在一起。每个项目将评估内容效度，评分的临床意义，对临床变化的反应，以及PROMIS儿科疼痛干扰，疲劳，心理压力体验和积极影响措施的纵向结构效度。COPR方法将根据PROMIS标准提高测量的心理测量和临床成熟度，并提供FDA要求的证据，以使PRO仪器符合临床试验的要求。COPR团队整合了：一个PROMIS研究小组，开发了20个现存PROMIS儿科措施中的10个，并在过去6年中帮助领导了PROMIS倡议;一个PCORI资助的临床数据研究网络（PEDSnet），在电子健康记录和PRO数据管理、质量评估和报告方面具有深厚的专业知识;一个全国儿科胃肠病学家网络，正在着手进行一项PCORI资助的克罗恩病儿童临床试验;一个由儿科肾脏科医生组成的全国网络，正在对患有慢性肾病的儿童进行NIH资助的队列研究;以及位于圣裘德儿童研究医院的全国最大的儿科肿瘤临床中心之一。COPR基础设施将：促进其三个研究项目之间的协调;标准化临床验证方法和信息资源;综合三个项目的结果;开发基于标准的仪器档案，以提高PROMIS的临床采用，并促进FDA对PROMIS措施作为临床试验终点的资格;以及支持与其他PEPR研究项目的合作。COPR进行了优化，以成功完成其三个研究项目，其基础设施可以随时扩展，以支持 信息学，统计和其他多个PEPR研究计划的行政需求。

项目成果

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

• DOI: 10.1016/j.jpeds.2021.10.053
• 发表时间: 2022-03
• 期刊: The Journal of pediatrics
• 作者: Miller TL;Schuchard J;Carle AC;Forrest CB;Kappelman MD;COMBINE Study Group
• 通讯作者: COMBINE Study Group